Note: This content was generated with the assistance of AI. All data points, PDUFA dates, and clinical trial milestones are sourced from publicly available company announcements, regulatory filings, and peer-reviewed literature as of March 2026.

Several notable regulatory and clinical milestones in ophthalmology are expected in the second quarter of 2026. A potential FDA decision on a thyroid eye disease (TED) therapy under Priority Review, phase 3 topline data for a subcutaneous TED candidate, an anticipated NDA filing for a sustained-release wet AMD implant, and the anticipated completion of a rolling biologics license application (BLA) for retinitis pigmentosa (RP) are all on the near-term horizon. This roundup includes programs with a publicly disclosed regulatory or clinical milestone expected through June 30, 2026, and is not exhaustive of all ophthalmology pipeline activity during this period.

Thyroid eye disease: Two programs with near-term milestones

PDUFA Date — June 30, 2026

Veligrotug (Viridian Therapeutics) · TED · IV Anti-IGF-1R Antibody

What it is. Veligrotug is an intravenously administered, fully human monoclonal antibody targeting the insulin-like growth factor-1 receptor (IGF-1R). By blocking IGF-1R signaling, it aims to reduce the orbital tissue inflammation and expansion that defines TED.

The regulatory picture. The FDA accepted Viridian’s BLA and granted Priority Review in December 2025, establishing a PDUFA target action date of June 30, 2026.¹ Priority Review shortens the standard 10-month review window to six months—a designation reserved for therapies that represent a significant improvement in the safety or effectiveness of treating a serious condition.¹ Veligrotug also holds Breakthrough Therapy Designation, granted in May 2025, making it the second FDA designation for the program in 2025.¹

Clinical basis. The BLA is built on data from the phase 3 THRIVE and THRIVE-2 trials, both of which met all primary and secondary end points. Veligrotug demonstrated rapid onset of treatment effect, clinically meaningful improvements in proptosis and diplopia, and durable responses, and was generally well-tolerated across both active and chronic TED populations.² The therapy uses a five-infusion, 12-week treatment course.¹

Commercial preparations. Viridian has stated commercial preparations are on track to support a planned mid-2026 US launch.² A Marketing Authorization Application was submitted to the European Medicines Agency in January 2026, on schedule.²

Phase 3 Topline Data — Q2 2026

Elegrobart / VRDN-003 (Viridian Therapeutics) · TED (Chronic) · Subcutaneous Anti-IGF-1R

What it is. Elegrobart (formerly VRDN-003) is a subcutaneous anti-IGF-1R antibody designed for self-administration at home via a low-volume autoinjector, with dosing intervals of every four or eight weeks, compared with the five-infusion, 12-week IV course used with veligrotug.

The trial. REVEAL-2 is a global phase 3 study evaluating elegrobart in patients with chronic TED. Topline data from REVEAL-2 are on track for Q2 2026, following the REVEAL-1 (active TED) readout targeted for Q1 2026.⁸ Both studies exceeded their target enrollment.³

Why it matters. If REVEAL-2 data are positive, Viridian plans to file a BLA for elegrobart by year-end 2026. The company has stated that commercial infrastructure being built for veligrotug’s anticipated launch is designed to lay the groundwork for a potential subsequent elegrobart rollout.³

Wet AMD: Sustained-release candidates advance toward regulatory filing

NDA Submission — Q2 2026 (anticipated)

AXPAXLI / OTX-TKI (Ocular Therapeutix) · Wet Age-Related Macular Degeneration · Sustained-Release IVT Implant

What it is. AXPAXLI (OTX-TKI) is an investigational, bioresorbable intravitreal hydrogel implant designed to continuously deliver axitinib — an FDA-approved multi-target tyrosine kinase inhibitor — for approximately 9 to 12 months per injection.

The NDA decision. In December 2025, Ocular Therapeutix announced plans to accelerate its NDA submission timeline for AXPAXLI in wet AMD, intending to file shortly after receiving positive year-one data from the SOL-1 phase 3 trial.⁴ The decision followed public statements from FDA leadership indicating potential acceptability of a single, well-controlled registrational trial for large indications like wet AMD.⁴

Positive SOL-1 data confirmed. On February 17, 2026, Ocular Therapeutix announced positive topline results from the SOL-1 phase 3 superiority trial. The company confirmed plans to submit an NDA based on SOL-1 data, subject to planned formal discussions with the FDA.⁵ The company has stated that, if approved, AXPAXLI would be the first TKI approved for wet AMD; Ocular Therapeutix has also noted that a superiority trial design, if accepted by the FDA, would be differentiated from currently approved agents.⁵

Regulatory path. Ocular intends to leverage the 505(b)(2) regulatory pathway, citing the fact that axitinib is already approved in non-ophthalmic indications, and holds a Special Protocol Assessment (SPA) agreement with the FDA for SOL-1.⁴

Retinitis pigmentosa: Rolling BLA completion anticipated

Rolling BLA — Full Submission Anticipated Early 2026

MCO-010 / sonpiretigene isteparvovec (Nanoscope Therapeutics) · Retinitis Pigmentosa · Gene-Agnostic Optogenetic Gene Therapy

What it is. MCO-010 is an ambient-light activatable, gene-agnostic optogenetic therapy delivered via a single intravitreal injection. Rather than targeting a specific genetic mutation, it reprograms surviving bipolar retinal cells to become photosensitive, making it applicable to the broad RP population across more than 100 known disease-associated genes and 1,000 distinct mutations, without requiring genetic testing.⁶

Regulatory milestone. Nanoscope initiated a rolling BLA submission to the FDA in July 2025, with full submission anticipated in early 2026. The application is eligible for Priority Review based on the program’s Fast Track designation.⁶ According to Nanoscope, this marks the first BLA submitted for a gene-agnostic gene therapy for retinal disease; the company has stated that MCO-010 has demonstrated the ability to improve visual function in RP patients with severe vision loss.⁶

Clinical evidence. The BLA is supported by data from the phase 2b/3 RESTORE trial (NCT04945772), in which MCO-010 met its primary endpoints for best corrected visual acuity in both dose groups at 52 weeks, with vision gains from baseline exceeding 0.3 LogMAR.⁶ Long-term follow-up through the REMAIN study demonstrated that vision gains were sustained at three years with no serious adverse events reported in treated eyes.⁷ Five-year safety data from the EXTEND study confirmed a consistent safety profile with no new signals.⁸

Trials entering enrollment in Q1–Q2 2026

Pivotal Phase Enrollment Initiating — Q1–Q2 2026

ATSN-201 (Atsena Therapeutics) · X-Linked Retinoschisis · AAV Gene Therapy

Background. ATSN-201 is a gene therapy candidate for X-linked retinoschisis (XLRS) leveraging AAV.SPR, a novel laterally spreading capsid designed to efficiently target photoreceptors in the central retina while avoiding the surgical risks of foveal detachment.⁹

What’s happening now. Atsena completed dosing in Part B of the ongoing phase 1/2/3 LIGHTHOUSE trial (NCT05878860) in January 2026, including all adult and pediatric cohorts, with no serious adverse events reported.⁹ Enrollment of the pivotal Part C cohort was planned for Q1 2026.⁹ The FDA agreed in July 2025 to expand the LIGHTHOUSE study into a continuous phase 1/2/3 trial, enabling it to serve as the pivotal study supporting a BLA submission anticipated in early 2028, and accelerating development by at least 1.5 years versus a separate phase 3 program.¹⁰

If approved. ATSN-201 would be the first gene therapy approved for XLRS, a condition affecting approximately 30,000 males in the US and EU with no currently approved treatments.⁹

Pivotal Phase 3 Trial Initiating — H1 2026

AURN001 / neltependocel (Aurion Biotech) · Corneal Endothelial Disease · Allogeneic Cell Therapy

What it is. AURN001 is an investigational, single-administration, allogeneic cell therapy combining human corneal endothelial cells (neltependocel) with a rho-kinase inhibitor (Y-27632). It is designed to repopulate the corneal endothelium in patients with corneal edema secondary to corneal endothelial dysfunction — a condition in which endothelial cells do not naturally regenerate, leading to progressive vision loss. Aurion Biotech, in which Alcon acquired a majority interest in 2025, has received both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designation from the FDA for this program.¹¹

Phase 1/2 results. Twelve-month data from the phase 1/2 CLARA trial (NCT06041256), presented at the American Academy of Ophthalmology Annual Meeting in October 2025, demonstrated a dose-dependent response. In the high-dose group, 65% of patients achieved a ≥15-letter gain in best corrected visual acuity at 12 months versus 0% in the comparator group (P < .0001). Mean BCVA improvement in the high-dose group was 12.5 letters, with a mean reduction in central corneal thickness of 23.2 μm. No graft rejections and no treatment-related serious adverse events were reported across any treatment group.¹¹

What’s ahead in Q2 2026. Based on these phase 1/2 findings, Aurion Biotech announced plans to initiate a pivotal phase 3 trial in the U.S. in the first half of 2026. The program is notable in that it addresses an indication — corneal endothelial disease — for which the current standard of care depends on donor tissue transplantation, a supply-constrained approach. AURN001 uses cultured donor cell lines, with each qualified donor potentially yielding a large number of therapeutic doses.¹¹

Looking ahead

Several programs in Q2 2026 carry regulatory or clinical decisions that will shape near-term treatment options in their respective indications. A potential FDA approval of veligrotug would introduce a second approved therapy for TED, while topline data from the REVEAL-2 study will help clarify the clinical profile of a subcutaneous alternative in the same class. An AXPAXLI NDA submission, if filed, would initiate FDA review of a sustained-release intravitreal TKI approach in wet AMD. For RP, the completion of the Nanoscope rolling BLA will determine whether a gene-agnostic optogenetic therapy advances to formal FDA review. Outcomes across these programs are pending and will be reported as they become available.

Clinicians are encouraged to monitor company investor relations pages and FDA.gov for real-time updates on PDUFA action dates, advisory committee meetings, and approval decisions.

References

1. Viridian Therapeutics announces BLA acceptance and priority review for veligrotug for the treatment of thyroid eye disease. News release. BusinessWire. December 22, 2025. Accessed March 24, 2026. https://www.businesswire.com/news/home/20251222159703/en/Viridian-Therapeutics-Announces-BLA-Acceptance-and-Priority-Review-for-Veligrotug-for-the-Treatment-of-Thyroid-Eye-Disease

2. Viridian Therapeutics highlights recent progress and reports fourth quarter and full year 2025 financial results. News release. BusinessWire. February 26, 2026. Accessed March 24, 2026. https://www.businesswire.com/news/home/20260226760846/en/Viridian-Therapeutics-Highlights-Recent-Progress-and-Reports-Fourth-Quarter-and-Full-Year-2025-Financial-Results

3. Viridian Therapeutics prepares for transformational 2026. News release. BusinessWire. January 6, 2026. Accessed March 24, 2026. https://www.businesswire.com/news/home/20260106202623/en/Viridian-Therapeutics-Prepares-for-Transformational-2026

4. Ocular Therapeutix announces plans to accelerate NDA submission timeline for AXPAXLI in wet AMD. News release. GlobeNewswire. December 8, 2025. Accessed March 24, 2026. https://www.globenewswire.com/news-release/2025/12/08/3201405/0/en/Ocular-Therapeutix-Announces-Plans-to-Accelerate-NDA-Submission-Timeline-for-AXPAXLI-in-Wet-AMD.html

5. Ocular Therapeutix reports positive results from landmark SOL-1 phase 3 superiority trial in wet AMD. News release. GlobeNewswire. February 17, 2026. Accessed March 24, 2026. https://www.globenewswire.com/news-release/2026/02/17/3239086/0/en/Ocular-Therapeutix-Reports-Positive-Results-from-Landmark-SOL-1-Phase-3-Superiority-Trial-in-Wet-AMD.html

6. Nanoscope Therapeutics initiates rolling submission of biologics license application to FDA for MCO-010, the first gene-agnostic therapy to treat retinitis pigmentosa. News release. PR Newswire. July 14, 2025. Accessed March 24, 2026. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-initiates-rolling-submission-of-biologics-license-application-to-fda-for-mco-010-the-first-gene-agnostic-therapy-to-treat-retinitis-pigmentosa-302503892.html

7. Nanoscope Therapeutics announces durable 3-year vision improvements from REMAIN study of MCO-010 in retinitis pigmentosa. News release. PR Newswire. October 22, 2025. Accessed March 24, 2026. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-announces-durable-3-year-vision-improvements-from-remain-study-of-mco-010-in-retinitis-pigmentosa-302590734.html

8. Nanoscope Therapeutics reports five-year safety results from phase 1/2a follow-up study of MCO-010 optogenetic therapy in retinitis pigmentosa. News release. PR Newswire. November 4, 2025. Accessed March 24, 2026. https://www.prnewswire.com/news-releases/nanoscope-therapeutics-reports-five-year-safety-results-from-phase-12a-follow-up-study-of-mco-010-optogenetic-therapy-in-retinitis-pigmentosa-302603355.html

9. Atsena completes dosing in part B of the phase I/II/III LIGHTHOUSE trial evaluating ATSN-201 to treat X-linked retinoschisis and announces plans for initiation of pivotal part C cohort. News release. GlobeNewswire. January 8, 2026. Accessed March 24, 2026. https://www.globenewswire.com/news-release/2026/01/08/3215273/0/en/Atsena-Completes-Dosing-in-Part-B-of-the-Phase-I-II-III-LIGHTHOUSE-Trial-Evaluating-ATSN-201-to-Treat-X-linked-Retinoschisis-and-Announces-Plans-for-Initiation-of-Pivotal-Part-C-Co.html

10. Atsena Therapeutics announces alignment with FDA on regulatory pathway to approval for ATSN-201 in X-linked retinoschisis (XLRS). News release. GlobeNewswire. July 9, 2025. Accessed March 24, 2026. https://www.globenewswire.com/news-release/2025/07/09/3112470/0/en/Atsena-Therapeutics-Announces-Alignment-with-FDA-on-Regulatory-Pathway-to-Approval-for-ATSN-201-in-X-Linked-Retinoschisis-XLRS.html

11. Aurion Biotech achieves all primary, secondary, and exploratory endpoints in AURN001 phase 1/2 CLARA trial at 12 months. News release. BusinessWire. October 18, 2025. Accessed March 24, 2026. https://www.businesswire.com/news/home/20251018009011/en/Aurion-Biotech-Achieves-All-Primary-Secondary-and-Exploratory-Endpoints-in-AURN001-Phase-12-CLARA-Trial-at-12-Months